How will FDA evaluate another case of muddled data?

On Wednesday the FDA will decide whether palovarotene, a drug for an ultra-rare genetic bone disease, merits approval. Patients already know that the drug is hardly a cure for fibrodysplasia ossificans progressiva, a disease that causes bones to grow out of control. But "even if it's not the perfect drug, to have something where there's data to show that it helps, that's just so important," one patient told STAT.

The treatment, made by the French drugmaker Ipsen, slows new bone formation — which can extend a patient's ability to perform daily tasks like dressing, eating, and using the bathroom independently. Advocates have fought tirelessly for its approval.

But ambiguous trial results could give regulators pause, even as patients clamor for the drug. A pivotal study showed that palovarotene failed to reduce bone formation — but when Ipsen reanalyzed the data, the drug showed some efficacy. This wasn't enough for European regulators, however, who rejected the drug earlier this year. But an FDA advisory committee in June largely endorsed the treatment, so patients are holding out hope.

Adam's Take

What's behind the recent surge in Apellis' stock?

A growing belief among investors that the serious safety issue that has derailed Syfovre, the company's eye disease treatment, is receding.

Declaring the crisis over is premature, but that could change with more uneventful weeks. Apellis has been a takeout target before, and may be again, at an acquisition price nearly twice its current market value.

Apellis shares rose 32% last week, with most of the gains coming Friday on heavy trading volume. The reason: There have been no new cases of retinal occlusive vasculitis, or ROV, reported in people receiving injections of Syfovre.

Read more of Adam Feuerstein's latest take on Apellis here.

gene therapy

When a $2.25 million drug doesn't work well enough

Zolgensma has been lauded as something of a miracle drug for patients with spinal muscular atrophy, a rare degenerative disease. But the $2.25 million gene therapy from Novartis hasn't been enough to slow the progression of the disease in some patients. A growing number are turning to additional drugs to supplement the work done by the costly gene therapy, Reuters writes.

Although Zolgensma has been seen as potentially curative, Novartis data show that almost a third of children in an ongoing study required further treatments. And if the gene therapy fails to work as hoped, it might be harder to justify the high prices it commands.

"The perception that Zolgensma is going to be a complete cure ... is not coming to fruition from the data we have seen over the last four years," Roger Hajjar, director of the Mass General Brigham Gene & Cell Therapy Institute, told Reuters. "Following the injection of Zolgensma you are basically going from a deadly disease to a more chronic disease state."

drug shortages

Survey: 99% of hospital pharmacists face drug shortages

Thanks to the ongoing shortages of prescription drugs, 85% of hospital pharmacists are rationing treatments, and 84% are choosing different dosages, a recent survey showed. About 59% are relying more heavily on compounding pharmacies.

The survey results underscore increasing concerns with manufacturing quality. Researchers at the Brookings Institution suggested that the U.S. government offer incentives to drugmakers to upgrade or expand their facilities through low-interest loans. The goal would be to create buffer inventories, particularly for generic sterile injectables that are in high demand at hospitals.

drug shortages

Tracing cisplatin's supply chain

It takes a global effort to create medicines that are in demand, and so any kink in the chain can contribute to the ongoing shortages. NBC News has outlined why the various steps in the supply chain to create the chemotherapy drug cisplatin can easily be compromised.

Platinum, the raw material needed for the drug, needs to be mined — and it's only available in a few countries, particularly South Africa and Russia. It needs to be refined in specialized facilities, and then from there, is typically sent to a manufacturing facility in either India or Germany. Turning platinum into cisplatin is quite difficult, and the process can be easily contaminated. It involves synthesizing the metal with ammonia and chlorine, which can be risky to work with.

The final step is "fill finish," which involves packaging, labeling, and readying the drugs for distribution. This tends to happen in FDA-approved facilities in Germany, Italy, India, the Netherlands, and the U.S. And even if a drugmaker wants to begin making a drug that's in short supply, it can't just jumpstart the process.

"It probably takes two years at least from the time that we know we need a drug to get FDA approval and get a factory up and get production started again," one researcher of these pharmaceutical supply chains told NBC.

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